Robert J. Hopkin, MD, on Assessing ST-920 for Fabry Disease
The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed Sangamo Therapeutics’ gene therapy, ST-920, and the phase 2 STAAR clinical trial.
Top News from WORLDSymposium 2023
Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.
Robert J. Hopkin, MD, on the Unmet Needs in Fabry Disease Treatment
The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed the limitations of the current standard of care for Fabry disease.
Gene Therapy Shows Promise in Infantile GM1 Gangliosidosis
Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.
Hematopoietic Cell Transplantation Leads to Improved Cardiac Function in MPSI
Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.
Fabry Disease Gene Therapy Demonstrates Clinically Meaningful Improvements
4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.
Fabry Disease Gene Therapy Demonstrates Favorable Impact in Phase 1/2 Trial
All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.
UX111 Shows Continued Dose-Dependent Efficacy as an MPS IIIA Treatment
Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.
Infantile GM1 Gangliosidosis Gene Therapy Demonstrates Safety and Dose-dependent Biomarker Changes
Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.
Gene Therapy Yields Neurodevelopmental Improvements in Children With MPSI
Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.
MPSII Gene Therapy Shows Some Signs of Durable Efficacy
Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.
Samiah Al-Zaidy, MD, on Gene Therapy for Infantile GM1 Gangliosidosis
Al-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.
Late-Onset Pompe Disease Gene Therapy Shows Promise in Phase 1/2 Study
Following treatment with AT845 gene therapy, 3 of 4 patients withdrew from their previous enzyme replacement therapy.
Arsa-cel Shows Favorable Risk/Benefit Profile in up to 11 Years of Follow-up Data
Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.
HSC Gene Therapy Demonstrates Improvements in Type 3 Gaucher Disease Case Study
The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.
Gaucher Disease HSC Gene Therapy Demonstrates Efficacy and Safety in Phase 1/2 Trial
Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.
Increased Motor Development Seen in First 2 Patients With Infantile Krabbe Treated With FBX-101
FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.
Stephanie Cherqui, PhD, on Bringing Attention to Cystinosis and Gene Therapy
The associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.
Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD
Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.
Cautious Optimism for GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.
Patient-Informed Study to Evaluate Hunter Syndrome Gene Therapy
The trial design was informed by both patient and physician perspectives.
Lysogene Gene Therapies Show Promise in GM1 Gangliosidosis and MPSIIIA
Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.
The Need for Analysis in Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.
The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.
First In-Human Trial to Assess Krabbe Disease Gene Therapy
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
Gene Therapy Well-Tolerated in Late-Onset Pompe Disease
Interim safety data from the FORTIS study were presented at WORLDSympsoium.
Achieving Developmental Milestones With GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium.
Molecular Analysis Elucidates Cell Therapy Destiny and Distribution
AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.
Gene Therapy Stabilizes Disease Progression in GM1 Gangliosidosis
Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.
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